We are thrilled with the progress across our pipeline, including the dosing of the first patient in our cystinosis program and receipt of orphan drug designation for our investigational gene therapy for Gaucher disease, commented Geoff MacKay, President and Chief Executive Officer of AVROBIO. In our Fabry program, we have now dosed eight patients across two clinical trials and we are on track to use our optimized lentiviral vector and a conditioning regimen utilizing therapeutic drug monitoring for the first time to dose a patient in our Phase 2 clinical trial for Fabry disease by the end of 2019. While our rapid expansion and early data have been exciting, we are humbled by the needs of the rare disease communities with whom we engage. They impress a sense of urgency on our work to deliver a new paradigm that we believe can supersede current treatment options and potentially provide patients freedom from a lifetime of disease.

Program Updates and Milestones

Third Quarter 2019 Financial Results

AVROBIO reported a net loss of $17.1 million for the third quarter of 2019 as compared to a net loss of $11.6 million for the comparable period in 2018. This increase was due to increased research and development expenses, as well as increased general and administrative expenses.

Research and development expenses were $13.0 million for the third quarter of 2019 as compared to $9.2 million for the comparable period in 2018. This increase was driven by increased program development activities related to the advancement of the Companys pipeline, as well as increased personnel-related costs resulting from an increase in employee headcount.

General and administrative expenses were $5.0 million for the third quarter of 2019 as compared to $3.0 million for the comparable period in 2018. This increase was primarily due to an increase in employee headcount, expenses associated with being a publicly traded company, including consulting expenses, and the impact of non-cash stock-based compensation.

As of September 30, 2019, AVROBIO had $206.4 million in cash and cash equivalents, as compared to $126.3 million in cash and cash equivalents as of December 31, 2018. The cash balance as of September 30, 2019 reflects the receipt of net proceeds of $129.5 million from the Companys July 2019 follow-on equity offering. Based on the Companys current operating plan, AVROBIO expects its cash and cash equivalents as of September 30, 2019 will enable the Company to fund its operating expenses and capital expenditure requirements into the second half of 2021.

About AVROBIO, Inc.

AVROBIO, Inc. is a leading, Phase 2 gene therapy company focused on the development of its investigational gene therapy, AVR-RD-01, in Fabry disease, as well as additional gene therapy programs in other lysosomal storage disorders including Gaucher disease, cystinosis and Pompe disease. The Companys plato platform includes a proprietary vector system, automated cell manufacturing solution and refined conditioning regimen deploying therapeutic drug monitoring. AVROBIO is headquartered in Cambridge, MA and has offices in Toronto, ON. For additional information, visit http://www.avrobio.com.

Forward-Looking Statements

This press release contains forward-looking statements, including statements made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. These statements may be identified by words such as aims, anticipates, believes, could, estimates, expects, forecasts, goal, intends, may, plans, possible, potential, seeks, will, and variations of these words or similar expressions that are intended to identify forward-looking statements. These forward-looking statements include, without limitation, statements regarding our business strategy, prospective products and goals, the therapeutic potential of our product candidates, the design, commencement, enrollment and timing of ongoing or planned clinical trials, clinical trial results, product approvals and regulatory pathways, the intended incentives conferred by orphan-drug designation, potential regulatory approvals and the timing thereof, expected benefits from the appointment of Ms. Verdin to the position of Chief Human Resources Officer and Ms. May to the position of Chief Commercial Officer, anticipated benefits of our gene therapy platform including potential impact on our commercialization activities, timing and likelihood of success, plans and objectives of management for future operations, future results of anticipated products, and the market opportunity for and anticipated commercial activities relating to our product candidates, and statements regarding the Companys financial and cash position and expected cash runway. Any such statements in this press release that are not statements of historical fact may be deemed to be forward-looking statements. Results in preclinical or early stage clinical trials may not be indicative of results from later stage or larger scale clinical trials and do not ensure regulatory approval. You should not place undue reliance on these statements, or the scientific data presented.

Any forward-looking statements in this press release are based on AVROBIOs current expectations, estimates and projections about our industry as well as managements current beliefs and expectations of future events only as of today and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, but are not limited to, the risk that any one or more of AVROBIOs product candidates will not be successfully developed or commercialized, the risk of cessation or delay of any ongoing or planned clinical trials of AVROBIO or our collaborators, the risk that AVROBIO may not realize the intended benefits of our gene therapy platform, including the features of our plato platform, the risk that our product candidates or procedures in connection with the administration thereof will not have the safety or efficacy profile that we anticipate, the risk that prior results, such as signals of safety, activity or durability of effect, observed from preclinical or clinical trials, will not be replicated or will not continue in ongoing or future studies or trials involving AVROBIOs product candidates, the risk that we will be unable to obtain and maintain regulatory approval for our product candidates, the risk that the size and growth potential of the market for our product candidates will not materialize as expected, risks associated with our dependence on third-party suppliers and manufacturers, risks regarding the accuracy of our estimates of expenses and future revenue, risks relating to our capital requirements and needs for additional financing, and risks relating to our ability to obtain and maintain intellectual property protection for our product candidates. For a discussion of these and other risks and uncertainties, and other important factors, any of which could cause AVROBIOs actual results to differ materially and adversely from those contained in the forward-looking statements, see the section entitled Risk Factors in AVROBIOs Quarterly Report on Form 10-Q for the quarter ended June 30, 2019, as well as discussions of potential risks, uncertainties and other important factors in AVROBIOs subsequent filings with the Securities and Exchange Commission. AVROBIO explicitly disclaims any obligation to update any forward-looking statements except to the extent required by law.